Abstract: This invention relates to polycomb-associated RNAs, libraries and fragments of those RNAs, inhibitory nucleic acids and methods and compositions for targeting RNAs, and methods of use thereof.
Type:
Grant
Filed:
March 17, 2016
Date of Patent:
January 26, 2021
Assignee:
The General Hospital Corporation
Inventors:
Jeannie T. Lee, Michael Rosenberg, Barry Kesner
Abstract: There is disclosed herein methods, uses and systems for the detection, diagnosis, prognosis, treatment or prevention of a disease or condition comprising cartilage degeneration in a subject that is in need thereof. The methods comprise the use, inhibition or measurement of at least one of miR-181 a-5p and miR-4454, in the subject.
Type:
Grant
Filed:
January 31, 2017
Date of Patent:
January 12, 2021
Assignee:
UNIVERSITY HEALTH NETWORK
Inventors:
Mohit Kapoor, Akihiro Nakamura, Raja Rampersaud
Abstract: The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the programmed cell death 1 ligand 1 (PD-L1) gene, and methods of using such RNAi agents to inhibit expression of a PD-L1 gene and methods of treating subjects having a PD-L1-associated disorder.
Abstract: Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.
Type:
Grant
Filed:
June 7, 2019
Date of Patent:
January 5, 2021
Assignee:
AVIDITY BIOSCIENCES, INC.
Inventors:
Andrew John Geall, Venkata Ramana Doppalapudi, David Sai-Ho Chu, Michael Caramian Cochran, Michael Hood, Beatrice Diana Darimont, Rob Burke, Yunyu Shi, Gulin Erdogan Marelius, Barbora Malecova
Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Down Syndrome Gene, in particular, by targeting natural antisense polynucleotides of a Down Syndrome Gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Down Syndrome Genes.
Abstract: Aspects of the present invention include the production and use of chemically modified RNAi agents (e.g., shRNAs) in gene silencing applications. The chemically modified RNAi agents disclosed herein have reduced immunostimulatory activity, increased serum stability, or both, as compared to a corresponding RNAi agent not having the chemical modification. Compositions containing chemically modified RNAi agents according to aspects of the present invention (including pharmaceutical compositions) and kits containing the same are also provided.
Type:
Grant
Filed:
October 5, 2017
Date of Patent:
December 22, 2020
Assignee:
SOMAGENICS, INC.
Inventors:
Qing Ge, Brian H. Johnston, Mark A. Behlke, Heini Ilves, Anne Dallas
Abstract: Provided herein are methods of treating NLRP3 inflammasome-associated diseases and disorders. Also, disclosed are methods for screening for agents useful in such methods.
Type:
Grant
Filed:
June 25, 2019
Date of Patent:
December 22, 2020
Assignee:
The Regents of the University of California
Abstract: Provided are a dsRNA construct of an orphan G-protein-coupled receptor GPR160 gene related to prostate cancer and the use thereof, wherein the dsRNA construct of the GPR160 gene and a composition thereof can prevent or treat prostate cancer.
Type:
Grant
Filed:
September 29, 2016
Date of Patent:
December 15, 2020
Assignee:
Shanghai Institute of Materia Medica, Chinese Academy of Sciences
Inventors:
Caihong Zhou, Xinchuan Dai, Mingwei Wang
Abstract: Disclosed are morpholino oligonucleotides that can be used to silence expression of MGMT, pharmaceutical compositions that include said morpholino oligonucleotides, and methods of using said morpholino oligonucleotides in the treatment of cancer, particularly methods that involve the use of radiation to deliver said morpholino oligonucleotides.
Type:
Grant
Filed:
July 25, 2019
Date of Patent:
December 8, 2020
Assignees:
UNITED STATES GOVERNMENT AS REPRESENTED BY THE DEPARTMENT OF VETERANS AFFAIRS, OREGON HEALTH & SCIENCE UNIVERSITY
Inventors:
Prakash Ambady, Jeffrey Wu, Edward Neuwelt
Abstract: The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.
Type:
Grant
Filed:
May 6, 2019
Date of Patent:
December 1, 2020
Assignee:
University of Massachusetts
Inventors:
Christian Mueller, Robert H. Brown, Jr.
Abstract: This invention provides methods of treating androgenetic alopecia (AGA), acne, rosacea, prostate cancer, and benign prostatic hypertrophy (BPH), comprising the step of contacting a subject with a compound or composition capable of decreasing prostaglandin D2 (PGD2) level or activity, a downstream signaling or receptor pathway thereof, or prostaglandin D2 synthase level or activity; methods of stimulating hair growth, comprising the step of contacting a subject with a compound or composition capable of increasing or decreasing the activity or level of a target gene of the present invention, or with a protein product of the target gene or an analogue or mimetic thereof; and methods of testing for AGA and evaluating therapeutic methods thereof, comprising measuring PGD2 levels.
Type:
Grant
Filed:
February 12, 2018
Date of Patent:
December 1, 2020
Assignee:
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
Abstract: The invention relates to double stranded ribonucleic acid (dsRNA) compositions targeting a glucokinase (GCK) gene, as well as methods of inhibiting expression of a glucokinase (GCK) gene, and methods of treating subjects having a glycogen storage disease (GSD), e.g., type Ia GSD.
Type:
Grant
Filed:
December 21, 2017
Date of Patent:
November 24, 2020
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Kevin Fitzgerald, David Erbe, Gregory Hinkle
Abstract: Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
Type:
Grant
Filed:
February 26, 2020
Date of Patent:
November 17, 2020
Assignee:
Ionis Pharmaceuticals, Inc.
Inventors:
Gene Hung, C. Frank Bennett, Susan M. Freier, Holly Kordasiewicz, Lisa Stanek, Don W. Cleveland, Seng H. Cheng, Lamya Shihabuddin
Abstract: The present invention relates to methods for treating a tumor, including a metastatic tumor, with TLR9 agonist in combination with an immune checkpoint inhibitor therapy.
Type:
Grant
Filed:
August 30, 2019
Date of Patent:
November 17, 2020
Assignee:
IDERA PHARMACEUTICALS, INC.
Inventors:
Sudhir Agrawal, Daqing Wang, Wayne Jiang
Abstract: Disclosed herein is a functionalized eyewear device that is adapted for collecting and analyzing disease biomarkers. Specifically, exemplified is a contact lens that has aptamer molecules associated therewith for binding to a specific biomarker (or biomarkers). The eyewear device is useful for detecting and diagnosing medical conditions.
Type:
Grant
Filed:
June 3, 2019
Date of Patent:
November 10, 2020
Assignee:
University of Central Florida Research Foundation, Inc.
Abstract: The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.
Type:
Grant
Filed:
February 14, 2019
Date of Patent:
November 10, 2020
Assignee:
Erasmus University Medical Center Rotterdam
Inventors:
Atze Jacobus Bergsma, Erik Van Der Wal, Wilhelmus Wenceslaus Matthias Pijnappel, Antje Tjitske Van Der Ploeg, Arnoldus Reuser
Abstract: In certain embodiments, the present disclosure provides compounds and methods of increasing the amount or activity of a target protein in a cell. In certain embodiments, the compounds comprise a translation suppression element inhibitor. In certain embodiments, the translation suppression element inhibitor is a uORF inhibitor. In certain embodiments, the uORF inhibitor is an antisense compound.
Type:
Grant
Filed:
November 16, 2015
Date of Patent:
November 3, 2020
Assignee:
Ionis Pharmaceuticals, Inc.
Inventors:
Stanley T. Crooke, Xue-hai Liang, Wen Shen
Abstract: The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal applications.
Type:
Grant
Filed:
February 26, 2018
Date of Patent:
October 27, 2020
Assignee:
Phio Pharmaceuticals Corp.
Inventors:
Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Pamela A. Pavco, James Cardia
Abstract: Described herein are aptamers capable of binding to growth differentiation factor 11 (GDF11) protein; compositions comprising a GDF11 binding aptamer with a GDF11 protein; and methods of making and using the same.
Type:
Grant
Filed:
November 23, 2015
Date of Patent:
October 20, 2020
Assignee:
SOMALOGIC, INC.
Inventors:
Urs Ochsner, Louis Green, Dom Zichi, Nebojsa Janjic
Abstract: The invention describes recombinant DNA sequences transcribed into RNA constructs capable of forming Virus Like Particles (VLPs) suitable for insect control applications. Specifically, the disclosure provides a method for controlling target insects comprising, transforming a microbial host with a first DNA sequence comprising a gene encoding a bacteriophage capsid protein and a second DNA sequence encoding an RNA transcript comprising at least one bacteriophage pac sequence coupled to an RNAi precursor sequence, inducing the microbial host to express the first and second DNA sequences, isolating virus-like-particles (VLPs) comprising the capsid protein and RNAi precursor from the microbial host, and contacting the isolated VLPs with the target insects.
Type:
Grant
Filed:
December 7, 2016
Date of Patent:
October 20, 2020
Assignee:
APSE, INC.
Inventors:
John L. Killmer, Juan Pedro Humberto Arhancet, Patrick D. McLaughlin